Rocket Pharmaceuticals Receives FDA RMAT Designation for Gene Therapy Targeting PKP2-Arrhythmogenic Cardiomyopathy
Friday, July 18, 2025
Rocket Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A601, its investigational gene therapy for plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM).
RP-A601 is an investigational gene therapy being developed to treat PKP2-related arrhythmogenic cardiomyopathy. It uses a recombinant adeno-associated virus (AAVrh74) to deliver a healthy version of the PKP2 gene. The therapy is administered as a single intravenous infusion and is designed to restore normal levels of plakophilin-2 protein in heart tissue. RP-A601 holds Fast Track and Orphan Drug designations in both the United States and Europe, recognising its potential as a treatment for a serious, rare condition with limited options.
PKP2-ACM is a genetic heart disease caused by mutations in the PKP2 gene. It leads to dangerous changes in the heart’s structure and function, including ventricular arrhythmias and a high risk of sudden cardiac death. Around 50,000 people in the US and Europe are estimated to be affected. Current treatments such as medications, implantable cardioverter defibrillators (ICDs), and ablation procedures may help manage symptoms but do not correct the underlying genetic defect. These approaches can also be associated with complications, highlighting the need for more effective and long-lasting therapies.
The designation is based on encouraging early results from a Phase 1 clinical trial.
RP-A601 is being studied as a potential one-time treatment using adeno-associated virus (AAV) technology. It aims to address the root genetic cause of PKP2-ACM, a rare and life-threatening inherited heart condition. The disease is marked by abnormal heart rhythms and a high risk of sudden cardiac death.
The RMAT designation is designed to speed up the development of therapies for serious conditions. It provides access to more frequent interactions with the FDA, and offers potential pathways for faster approval based on early clinical data.
Preliminary results from the trial presented at a recent scientific meeting showed that three adult patients treated with RP-A601 experienced increased levels of PKP2 protein. Improvements were also observed in structural heart tissue and in clinical measures such as heart function, arrhythmia control, and quality of life. The treatment was generally well tolerated, with no serious safety concerns reported.
Rocket Pharmaceuticals continues to advance RP-A601 as part of its broader pipeline of gene therapies focused on rare and inherited cardiovascular conditions.
Source: rocketpharma.com
